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EP 3 758 719 A1

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(43) Date of publication: 06.01.2021 Bulletin 2021/01 (21) Application number: 19761695.6 (22) Date of filing: 01.03.2019

(51) International Patent Classification (IPC):  A61K 35/12(2015.01) C07K 14/72(2006.01) C12N 5/071(2010.01) A61P 3/08(2006.01) C12N 5/00(2006.01) C12N 5/077(2010.01) (86) International application number: PCT/US2019/020430 (87) International publication number: WO 2019/169351 (06.09.2019 Gazette 2019/36)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 02.03.2018 US 201862637923 P (71) Applicant: Vertex Pharmaceuticals Inc. Boston, MA 02210 (US)

(72) Inventors: PAGLIUCA, Felicia Cambridge, Massachusetts 02142 (US) HARB, George Cambridge, Massachusetts 02142 (US) GURTLER, Mads Cambridge, Massachusetts 02142 (US) GAGLIA, Jason Cambridge, Massachusetts 02142 (US) POH, Yeh-Chuin Cambridge, Massachusetts 02142 (US) YE, Lillian Cambridge, Massachusetts 02142 (US) THIEL, Austin Cambridge, Massachusetts 02142 (US) YASIN, Jihad Cambridge, Massachusetts 02142 (US) THOMPSON, Evrett Cambridge, Massachusetts 02142 (US) (74) Representative: J A Kemp LLP  14 South Square Gray's Inn London WC1R 5JJ London WC1R 5JJ (GB)